How are you taking part in this consultation?

You will not be able to change how you comment later.

You must be signed in to answer questions

  • Question on Consultation

    Has all of the relevant evidence been taken into account?

  • Question on Consultation

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

  • Question on Consultation

    Are the recommendations sound and a suitable basis for guidance to the NHS?

  • Question on Consultation

    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of age, disability, gender reassignment, pregnancy and maternity, race, religion or belief, sex or sexual orientation?

Avapritinib for treating inadequately controlled moderate to severe indolent systemic mastocytosis

Open for comments Open until   Request commenting lead permission

We have updated this service so that members of the same organisation can now collaborate on a joint online response.

Read our blog to learn more.

1 Recommendations

1.1

Avapritinib should not be used to treat moderate to severe indolent systemic mastocytosis that is inadequately controlled on symptomatic treatment in adults.

1.2

This recommendation is not intended to affect treatment with avapritinib that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop.

What this means in practice

These are NICE's draft recommendations. If these recommendations become final, avapritinib would not be required to be funded and should not be used routinely in the NHS in England for the condition and population in the recommendations.

This is because there is not enough evidence to suggest avapritinib offers value for money in this population.

Why the committee made these recommendations

Standard treatment for moderate to severe indolent systemic mastocytosis is symptomatic treatment (also called symptom-directed therapy). This aims to manage symptoms. Avapritinib aims to treat the underlying condition.

Clinical trial evidence shows that avapritinib reduces the severity of symptoms compared with symptom-directed therapy alone.

There are uncertainties in the economic model. It is unclear if the population and

definition of treatment response used in the model are the same as in NHS practice. And there are uncertainties about some of the assumptions, including that the modelling:

  • did not account for people having a higher dose of avapritinib in the trial

  • did not fully account for missing data on quality of life

  • did not use evidence to estimate the differences in costs when there was a response to treatment.

Because of the uncertainties in the economic model, it is not possible to determine the most likely cost-effectiveness estimates for avapritinib. So, avapritinib should not be used.